Study Design

Intravenously administered 900mg once per week for 4 weeks, 1200 mg on week 5 and then 1200mg every 2 weeks thereafter.

Primary Outcomes

Measure

Assess the effect of eculizumab on thrombotic microangiopathy (TMA).

time frame:
Through 26 weeks

Secondary Outcomes

Measure

Additional efficacy endpoints related to manifestations of TMA.

time frame:
Through 26 weeks

Overall safety and tolerability of eculizumab

time frame:
Through 26 weeks

Pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in patients with aHUS.

time frame:
Through 26 weeks

Eligibility Criteria

Male or female participants from 12 years up to 18 years old.

Inclusion Criteria:
1. Male or female patients from 12 and up to 18 years of age who have been diagnosed
with Atypical Hemolytic-Uremic Syndrome (aHUS).
2. Patients must be receiving PT for aHUS.
3. Platelet Count Pre-PT Baseline Set-Point (collected immediately prior to the
Qualifying PT Episode) is within 75% of the average of the pre-PT platelet counts
collected at Screening and during the Observation Period.
4. Diagnosis of aHUS
5. Lactate dehydrogenase (LDH) level ≥ ULN.
6. Creatinine level ≥ ULN for age.
7. Sexually active women of childbearing potential must be practicing an effective,
reliable and medically acceptable contraceptive regimen during the entire duration of
the study, including the follow-up period.
8. Patient's parents/legal guardian must be willing and able to give written informed
consent and patient must be willing to give written informed assent.
9. Able and willing to comply with study procedures.
Exclusion Criteria:
1. ADAMTS-13 inhibitor or deficiency (i.e., ADAMTS-13 activity <5%) as measured at the
screening visit.
2. Malignancy.
3. Typical HUS (Shiga toxin +).
4. Known HIV infection.
5. Identified drug exposure-related HUS.
6. Infection-related HUS.
7. Presence or suspicion of active and untreated systemic bacterial infection that, in
the opinion of the Investigator confounds an accurate diagnosis of aHUS or impedes
the ability to manage the aHUS disease.
8. Pregnancy or lactation.
9. Unresolved meningococcal disease.
10. Known Systemic Lupus Erythematosus (SLE) or antiphospholipid antibody positivity or
syndrome.
11. Any medical or psychological condition that, in the opinion of the investigator,
could increase the patient's risk by participating in the study or confound the
outcome of the study.
12. Patients receiving IVIg or Rituximab therapy.
13. Patients receiving other immunosuppressive therapies such as steroids, mTOR
inhibitors or FK506 inhibitors are excluded unless: [1] part of a post-transplant
anti-rejection regime, [2] patient has confirmed anti-CFH antibody requiring
immunosuppressive therapy and [3] dose of such medications have been unchanged for at
least 4 weeks prior to the screening period.
14. Patients receiving Erythrocyte Stimulating Agents (ESAs) unless already on a stable
dose for at least 4 weeks prior to the screening period.
15. Participation in any other investigational drug trial or exposure to other
investigational agent, device, or procedures beginning 4 weeks prior to screening and
throughout the entire trial.

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Trial Details

Related Tags

A monoclonal antibody used to prevent red blood cells from being destroyed in patients with paroxysmal nocturnal hemoglobinuria (PNH), a red blood cell disorder. Eculizumab blocks a protein in the blood that causes the breakdown of red blood cells.